One Gene, Two Diseases: the Pathologic Role of IGLV1-44 in AL Amyloidosis and POEMS

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-99
SponsorFondazione IRCCS Policlinico San Matteo di Pavia

About this trial

By detailed sequence analysis and subsequent biophysical characterization of prototypic light chains, this project aims to identify sequence fingerprints in IGLV1-44 light chains leading to AL amyloidosis and POEMS syndrome. This understanding might help improve the risk stratification and early diagnosis of patients overexpressing pathologic IGLV1-44 LCs. Moreover, the development of nanobodies efficient in recognizing and stabilizing IGLV1-44 light chains which exert direct toxicity in cardiac AL amyloidosis and POEMS syndrome might form the basis for future development of therapeutic agents capable of counteracting IGLV1-44 light chain proteotoxicity.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Biopsy-proven diagnosis of systemic AL amyloidosis, POEMS syndrome or multiple myeloma

Planned peripheral blood sampling +/- bone marrow aspiration

Age > 18 years

Willingness to allow use of clinical data and diagnostic leftovers of clinical specimens for research purposes through signing a written informed consent.

Disqualifiers

Undefined monoclonal gammopathy or non-AL amyloidosis

Patients fulfilling the criteria for complete hematologic response after anti-clonal therapy

Age <18 years

Failure to show willingness to allow use of clinical data and diagnostic leftovers of clinical specimens for research purposes.

Trial population

Patients with biopsy-proven diagnosis of systemic AL amyloidosis, POEMS syndrome or multiple myeloma

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

No trial groups listed

Trial outcomes

Primary outcomes

1

identify sequence fingerprints in IGLV1-44 light chains leading to AL amyloidosis and POEMS syndrome

By detailed sequence analysis and subsequent biophysical characterization of prototypic light chains, this project aims to identify sequence fingerprints in IGLV1-44 light chains leading to AL amyloidosis and POEMS syndrome.

Time frame
two years

Secondary outcomes

Other outcomes

Sponsors and contacts

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